Isis Pharma greatly narrows loss

CARLSBAD  Isis Pharmaceuticals said Thursday it closed 2012 with a quarterly loss of $2.6 million, or 3 cents per share, compared with a loss of $20 million, or 20 cents per share, for the same quarter a year ago.

An $18.4 million gain from its investment in Regulus, a San Diego-based biotech, and a related $9.1 million tax benefit were the main causes of the reduced loss, Isis said. The Carlsbad company had $374 million in cash and equivalents at the end of the year, compared with $344 million in the year-ago quarter.

This year is shaping up as a strong one for Isis, officials said in a conference call, as sales approach for its flagship drug Kynamro and the company brings in cash from its numerous drug development partnerships.

In January, Isis and its partner Genzyme received long-awaited U.S. approval to begin selling Kynamro, which treats a genetically caused disease that produces extremely high cholesterol levels. Isis received $25 million from Genzyme as a milestone payment for that approval. Genzyme is a unit of Paris-based Sanofi.

In February, Isis received a $7.5 million milestone payment from GlaxoSmithKline for beginning a clinical trial of a drug called Isis-TTRRx to treat another genetic disease, transthyretin amyloidosis. The disease damages peripheral nerves and the heart, leading to heart failure and death.

Both drugs use Isis' gene-blocking antisense technology, which stops production of disease-causing proteins. The approval of Kynamro is proof that the technology can produce a significant drug. The drug may generate more than $400 million in peak annual sales by 2020, according to one analyst estimate.

"Having Kynamro approved was a great way to start the year," said Isis CEO Stanley T. Crooke in a conference call. "It is the beginning of what we hope will be many, many more Isis drugs to reach the market."

Crooke said Kynamro's approval demonstrates that Isis' more potent second generation antisense technology is a "productive platform" for drug discovery and development, alongside the more traditional technologies of small molecule and protein drugs.

Isis has now generated 28 drugs in development, Crooke said.

"We plan to continue this growth by adding three to five new drugs every year," he said.

The Kynamro approval indicates that Isis is on the right track, said John Reed, the outgoing CEO of the Sanford-Burnham Medical Research Institute. In April, Reed will become leader of Swiss drug giant Roche's pharmaceutical research and early development group.

"It's an indication that platform has started to mature to the point where there's really a there there," Reed said, speaking Wednesday at a biotechnology conference held by Biocom, the San Diego life science trade group. "That technology, I think, has turned the corner."